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Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochloride

BACKGROUND: Sapropterin dihydrochloride effectively lowers plasma phenylalanine (Phe) for at least a third of phenylketonuria (PKU) patients, with potential for increased dietary Phe tolerance and decreased medical food requirement. OBJECTIVE: To investigate long-term quality of life (QOL) in patien...

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Detalles Bibliográficos
Autores principales:	Douglas, Teresa D, Ramakrishnan, Usha, Kable, Julie A, Singh, Rani H
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2013
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3880979/ https://www.ncbi.nlm.nih.gov/pubmed/24373161 http://dx.doi.org/10.1186/1477-7525-11-218

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